A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

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daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period.The Diamond Blackfan anemia arm was closed due to lack of accrual.

daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period. The Diamond Blackfan anemia arm was closed due to lack of accrual.

Daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions to subjects diagnosed with moderate aplastic anemia (MAA), pure red cell aplasia (PRCA), Diamond Blackfan anemia (DBA), relapse and refractory severe aplastic anemia (SAA) will receive treatment. The Diamond Blackfan anemia arm was closed due to the lack of accrual. The hematologic response will be evaluated at 3 months.

A complete hematologic response will be considered an achievement of normal blood counts. A partial response was defined as any response less than a complete response. The primary endpoint was a hematologic response in at least one affected peripheral blood count parameter, as determined by 3 separate measurements in the first 12 weeks after completion of the infusion.

Time Frame

3 months

Outcome Measure Data Outcome Measure Data

Analysis Population Description

Analysis Population Description

The daclizumab hematologic response was evaluated for subjects diagnosed with moderate aplastic anemia (MAA) and pure red cell aplasia (PRCA). The Diamond Blackfan arm was closed due to lack of accrual.

daclizumab, 1 mg/kg of body weight, will be given for a total of 5 intravenous infusions. The subjects diagnosed with moderate aplastic anemia, pure red cell aplasia, Diamond Blackfan anemia, relapse and refractory severe aplastic anemia will receive treatment. The subjects will be seen and receive the daclizumab infusion biweekly during the treatment period. The Diamond Blackfan anemia arm was closed due to lack of accrual.

All-Cause Mortality

Daclizumab Hematologic Response

Affected / at Risk (%)

Total

--/--

Serious Adverse Events Serious Adverse Events

Daclizumab Hematologic Response

Affected / at Risk (%)

# Events

Total

9/100 (9.00%)

Blood and lymphatic system disorders

polycythemia *

1/100 (1.00%)

1

Cardiac disorders

angina *

1/100 (1.00%)

1

Hepatobiliary disorders

elevated liver function tests *

1/100 (1.00%)

1

Immune system disorders

infection *

2/100 (2.00%)

2

Infections and infestations

sinusitis *

1/100 (1.00%)

1

gastroenteritis * [1]

1/100 (1.00%)

1

Neoplasms benign, malignant and unspecified (incl cysts and polyps)

hepatocellular carcinoma * [2]

1/100 (1.00%)

1

myeloma *

1/100 (1.00%)

1

Renal and urinary disorders

renal failure *

1/100 (1.00%)

1

*

Indicates events were collected by non-systematic assessment

[1]

wound- infectious

[2]

Herpes zoster

Other (Not Including Serious) Adverse Events Other (Not Including Serious) Adverse Events